Baseline demographic characteristics (prior systemic therapy pooled efficacy and prior systemic therapy pooled safety populations)
| . | Prior systemic therapy pooled efficacy population (n = 31) . | Prior systemic therapy pooled safety population (n = 53) . |
|---|---|---|
| Age, median years (range) | 68 (37-82) | 69 (31-86) |
| Male | 21 (68) | 35 (66) |
| Female, n (%) | 10 (32) | 18 (34) |
| Race, white n (%) | 31 (100) | 47 (89) |
| ECOG performance status, n (%) | ||
| 0-1 | 20 (65) | 36 (68) |
| 2-3 | 11 (35) | 17 (32) |
| AdvSM subtype (central assessment), n (%) | ||
| ASM | 1 (3) | 6 (11) |
| SM-AHN∗ | 22 (71) | 34 (64) |
| MCL | 8 (26) | 13 (25) |
| KIT D816V mutation by central assay, n (%) | 28 (90) | 49 (92) |
| ≥1 S/A/R mutation per central assay, n (%) | 13 (42) | 20 (38) |
| BM MC burden, median % (range) | 60.0 (10.0-95.0) | 50.0 (1.0-95.0) |
| Serum tryptase level, median ng/mL (range) | 334.0 (23.8-1600.0) | 312.0 (19.9-1600.0) |
| Spleen volume, median mL (range) | 781.6 (298.5-2270.0) | 781.6 (44.2-2600.8) |
| KIT D816V VAF in peripheral blood, median % (range) | 13.4 (0-45.3) | 18.9 (0-47.5) |
| Number of prior systemic therapies, n (%) | ||
| 1 | 18 (58) | 29 (55) |
| ≥2 | 13 (42) | 24 (45) |
| Prior systemic therapy, n (%)† | ||
| Azacitidine | 2 (6) | 3 (6) |
| Cladribine | 4 (13) | 10 (19) |
| Hydroxyurea | 3 (10) | 4 (8) |
| Interferon‡ | 4 (13) | 8 (15) |
| Midostaurin | 23 (74) | 43 (81) |
| Other | 10 (32) | 16 (30) |
| Brentuximab vedotin | 0 | 0 |
| Dasatinib | 1 (3) | 5 (9) |
| Decitabine | 1 (3) | 1 (2) |
| Imatinib | 5 (16) | 7 (13) |
| Investigational antineoplastic drugs | 2 (6) | 3 (6) |
| Nilotinib | 0 | 2 (4) |
| Protein kinase inhibitors (unspecified) | 0 | 1 (2) |
| Purine analogs | 0 | 1 (2) |
| Radiotherapy | 0 | 0 |
| alloHSCT | 1 (3) | 1 (2) |
| Thalidomide | 1 (3) | 1 (2) |
| Reason for discontinuation of prior therapy, n (%) | ||
| Completed scheduled cycles of treatment | 0 | 2 (4) |
| Disease progression§ | 18 (58) | 23 (43) |
| Toxicity | 7 (23) | 14 (26) |
| Otherǁ | 6 (19) | 14 (26) |
| Best response to most recent prior therapy, n (%) | ||
| CR | 0 | 0 |
| PR | 6 (19) | 11 (21) |
| CI | 3 (10) | 6 (11) |
| SD | 8 (26) | 13 (25) |
| PD | 5 (16) | 6 (11) |
| Otherǁ | 9 (29) | 17 (32) |
| Median duration on most recent prior therapy, months (range) | 7.9 (0-121.8) | 8.0 (0-121.8) |
| . | Prior systemic therapy pooled efficacy population (n = 31) . | Prior systemic therapy pooled safety population (n = 53) . |
|---|---|---|
| Age, median years (range) | 68 (37-82) | 69 (31-86) |
| Male | 21 (68) | 35 (66) |
| Female, n (%) | 10 (32) | 18 (34) |
| Race, white n (%) | 31 (100) | 47 (89) |
| ECOG performance status, n (%) | ||
| 0-1 | 20 (65) | 36 (68) |
| 2-3 | 11 (35) | 17 (32) |
| AdvSM subtype (central assessment), n (%) | ||
| ASM | 1 (3) | 6 (11) |
| SM-AHN∗ | 22 (71) | 34 (64) |
| MCL | 8 (26) | 13 (25) |
| KIT D816V mutation by central assay, n (%) | 28 (90) | 49 (92) |
| ≥1 S/A/R mutation per central assay, n (%) | 13 (42) | 20 (38) |
| BM MC burden, median % (range) | 60.0 (10.0-95.0) | 50.0 (1.0-95.0) |
| Serum tryptase level, median ng/mL (range) | 334.0 (23.8-1600.0) | 312.0 (19.9-1600.0) |
| Spleen volume, median mL (range) | 781.6 (298.5-2270.0) | 781.6 (44.2-2600.8) |
| KIT D816V VAF in peripheral blood, median % (range) | 13.4 (0-45.3) | 18.9 (0-47.5) |
| Number of prior systemic therapies, n (%) | ||
| 1 | 18 (58) | 29 (55) |
| ≥2 | 13 (42) | 24 (45) |
| Prior systemic therapy, n (%)† | ||
| Azacitidine | 2 (6) | 3 (6) |
| Cladribine | 4 (13) | 10 (19) |
| Hydroxyurea | 3 (10) | 4 (8) |
| Interferon‡ | 4 (13) | 8 (15) |
| Midostaurin | 23 (74) | 43 (81) |
| Other | 10 (32) | 16 (30) |
| Brentuximab vedotin | 0 | 0 |
| Dasatinib | 1 (3) | 5 (9) |
| Decitabine | 1 (3) | 1 (2) |
| Imatinib | 5 (16) | 7 (13) |
| Investigational antineoplastic drugs | 2 (6) | 3 (6) |
| Nilotinib | 0 | 2 (4) |
| Protein kinase inhibitors (unspecified) | 0 | 1 (2) |
| Purine analogs | 0 | 1 (2) |
| Radiotherapy | 0 | 0 |
| alloHSCT | 1 (3) | 1 (2) |
| Thalidomide | 1 (3) | 1 (2) |
| Reason for discontinuation of prior therapy, n (%) | ||
| Completed scheduled cycles of treatment | 0 | 2 (4) |
| Disease progression§ | 18 (58) | 23 (43) |
| Toxicity | 7 (23) | 14 (26) |
| Otherǁ | 6 (19) | 14 (26) |
| Best response to most recent prior therapy, n (%) | ||
| CR | 0 | 0 |
| PR | 6 (19) | 11 (21) |
| CI | 3 (10) | 6 (11) |
| SD | 8 (26) | 13 (25) |
| PD | 5 (16) | 6 (11) |
| Otherǁ | 9 (29) | 17 (32) |
| Median duration on most recent prior therapy, months (range) | 7.9 (0-121.8) | 8.0 (0-121.8) |
Percentages referring to patient numbers have been rounded to whole numbers and may not add up to 100%.
BM, bone marrow; ECOG, Eastern Oncology Cooperative Group; MC, mast cell; MPN-U, myeloproliferative neoplasm - unclassified; SD, stable disease; VAF, variant allele fraction.
Subtypes of AHN were: Prior systemic therapy pooled efficacy population: CEL, n = 3; CMML, n = 9; MDS, n = 3; MDS/MPN-U, n = 4; MPN, n = 1; and other, n = 2. Prior systemic therapy pooled safety population: CEL, n = 3; CMML, n = 15; MDS, n = 3; MDS/MPN-U, n = 7; MPN, n = 2; and other, n = 4.
Patients may have received >1 prior systemic therapy.
Includes pegylated interferons.
Includes patients who discontinued due to PD/relapse/refractory disease.
“Other” includes combined data for “other response,” unknown, and missing data.